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CAR-T产品医保准入风险分担协议的国际经验及启示 Δ

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李 伟 1，2＊ ，覃胡莉 ，丁锦希 ，李佳明 ，夏启瑞（1.中国药科大学国际医药商学院，南京 211198；2.中国药科
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大学医药市场准入政策研究中心，南京 211198）
中图分类号 R956；F840.684 文献标志码 A 文章编号 1001-0408（2021）24-2957-06
DOI 10.6039/j.issn.1001-0408.2021.24.03

摘 要 目的：借鉴国外已上市嵌合抗原受体T淋巴细胞（CAR-T）产品在医保准入中签订风险分担协议的经验，为我国医保部门
的相关决策提供参考。方法：以目前已上市的 CAR-T 产品在英国、法国、意大利、德国等 4 个样本国家的 9 份风险分担协议为样
本，从协议类型、监测指标、数据收集方式、协议周期、支付条件和支付方式等6个维度剖析CAR-T产品医保支付的国际经验，并为
该类产品在我国的医保准入提出建议。结果与结论：4个样本国家普遍与医药企业签订了医保准入风险分担协议（包括经济导向
协议和疗效导向协议），通过使用数据收集系统或临床研究数据来收集患者个体的疾病进展、无进展生存期等指标数据，从而监测
CAR-T产品的疗效与安全性；根据不同协议类型，确定协议周期与支付条件，通过使用“医保先行支付”和“医药企业垫付”2种方
式并结合“分期付款”来进行风险控制。解决“疗效不确定性”所带来的医保基金风险是CAR-T产品准入的核心问题，引入风险分
担协议可能是解决这一问题的途径，而科学设计风险分担协议的各要素则是确保协议可操作的前提条件。
关键词 嵌合抗原受体T淋巴细胞产品；医保准入；风险分担协议；国际经验；启示

Inspiration from International Experience on Risk Sharing Agreements of Medical Insurance Access for
CAR-T Products
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LI Wei ，QIN Huli ，DING Jinxi ，LI Jiaming ，XIA Qirui（1. School of International Pharmaceutical Business，
China Pharmaceutical University，Nanjing 211198，China；2. Center for Pharmaceutical Market Access Policy
Research，China Pharmaceutical University，Nanjing 211198，China）
ABSTRACT OBJECTIVE：To learn from the experience of foreign listed chimeric antigen receptor T lymphocyte （CAR-T）
products in signing risk sharing agreements in medical insurance access，so as to provide references for relevant decisions of
medical insurance departments in China. METHODS：Taking 9 risk sharing agreements of CAR-T products marketed in the United
Kingdom，France，Italy and Germany as samples，the international experience of medical insurance payment of CAR-T products
were analyzed from six dimensions，such as agreement types，monitoring indicators，data collection methods，agreement periods，
payment conditions and payment methods. Some suggestions were put forward for the medical insurance access of these products in
China. RESULTS & CONCLUSIONS：Four sample countries generally signed risk sharing agreements of medical insurance access
（financial agreement and performance-based agreement）with pharmaceutical enterprises；the indicators such as progressive disease
and progression-free survival were collected by using data collection system or clinical research data，so as to monitor the efficacy
and safety of CAR-T products. The agreement periods and payment conditions were determined according to different agreement
types；“medical insurance advance payment”or“pharmaceutical enterprise advance payment”combined with“staged payments”
were adopted for risk control. Solving the risk of medical insurance funds caused by“efficacy uncertainty”is the core issue of
CAR-T product access. The induction of risk sharing agreements may be the way to solve this problem，and the scientific design of
the various elements of risk sharing agreements is a prerequisite to ensure that the agreement is operational.
KEYWORDS Chimeric antigen receptor T lymphocyte products； Medical insurance access； Risk sharing agreements；
International experience；Inspiration

Δ 基金项目：江苏省教育厅 2019 年度高校哲学社会科学研究一 嵌合抗原受体 T 淋巴细胞（chimeric antigen recep-
般项目（No.2019SJA0063）；中国药科大学“双一流”科技创新团队项目 tor T lymphocyte，CAR-T）疗法是一种全新的针对患者
（No.CPU2018GY41） 基因缺陷的个体化细胞免疫治疗方法。在 2012 年美国
＊讲师，博士研究生。研究方向：医药市场准入与医疗保障政
宾夕法尼亚大学的一项临床试验中，1 例 5 岁的急性淋
策。E-mail：cpuliwei@163.com
巴细胞白血病（acute lymphocytic leukemia，ALL）患儿首
# 通信作者：教授，博士生导师，博士。研究方向：医药政策法规、
医药市场准入与药物经济学。电话：025-86185287。E-mail：13605152326@ 次尝试了 CAR-T 疗法，其体内的肿瘤细胞经 CAR-T 治
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163.com 疗后彻底消失，并且无癌生存超过9年 。根据Clinical-

中国药房 2021年第32卷第24期 China Pharmacy 2021 Vol. 32 No. 24 ·2957 ·